Development and Validation of a Disability Severity Index for CMT

Charcot Marie Tooth Disease Clinical Trial

— 6604  

Official title:

Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01455623
• Other study ID #: INC-6604
• Status: Completed
• Phase: N/A
• First received: October 17, 2011
• Last updated: March 25, 2014
• Start date: June 2011
• Est. completion date: March 2014

Study information

• Verified date: March 2014
• Source: University of Iowa
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial Summary

It is necessary for outcome measures to accurately reflect the state of health of a person in order for clinical trials to show benefit. The most commonly used outcome measure for Charcot Marie Tooth Disease (CMT) is the CMT Neuropathy Score, which uses cutoffs of points designated as mild (0-10 points), moderate (11-20) or severe (21-36). These terms are arbitrary. This study is looking to base mild, moderate, and severe on what both people affected with CMT and those who provide for people with CMT consider appropriate.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 291
• Est. completion date: March 2014
• Est. primary completion date: March 2014
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 8 Years and older

Eligibility

Inclusion Criteria:

• patients with CMT ages 8-100,

• health care professionals who take care of patients with CMT.

• Eligible patients will be patients that have joined the INC RDCRN Contact Registry. These will be patients or guardians with known or probable inherited neuropathies.

• Eligible health care professionals who participate in the care of patients with inherited neuropathies (physicians, genetic counselors, physical and occupational therapists etc.) attending the 4th International CMT Consortium to be held in Potomac Maryland June 29-July 1, 2011.

Exclusion Criteria:

• Does not read or speak English.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Charcot Marie Tooth Disease
• Charcot-Marie-Tooth Disease
• Hereditary Sensory and Motor Neuropathy
• Nerve Compression Syndromes
• Tooth Diseases

Locations

Country	Name	City	State
United States	University of Iowa	Iowa City	Iowa

Sponsors (3)

Lead Sponsor	Collaborator
University of Iowa	Muscular Dystrophy Association, National Institute of Neurological Disorders and Stroke (NINDS)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT		1 year	No
Secondary	Develop a Disability Severity Index including items with high patient-physician concordance		1 year	No